Article
Alpha-1 Antitrypsin Augmentation and the Liver Phenotype of Adults With Alpha-1 Antitrypsin Deficiency (Genotype Pi∗ZZ).
Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association - 1 Feb 2024
Fromme Malin, Hamesch Karim, Schneider Carolin V, Mandorfer Mattias, Pons Monica, Thorhauge Katrine H, Pereira Vitor, Sperl Jan, Frankova Sona, Reichert Matthias C, Benini Federica, Burbaum Barbara, Kleinjans Moritz, Amzou Samira, Rademacher Laura, Bewersdorf Lisa, Verbeek Jef, Nevens Frederik, Genesca Joan, Miravitlles Marc, Nuñez Alexa, Schaefer Benedikt, Zoller Heinz, Janciauskiene Sabina, Waern Johan, Oliveira António, Maia Luís, Simões Carolina, Mahadeva Ravi, Fraughen Daniel D, Trauner Michael, Krag Aleksander, Lammert Frank, Bals Robert, Gaisa Nadine T, Aigner Elmar, Griffiths William J, Denk Helmut, Teumer Alexander, McElvaney Noel G, Turner Alice M, Trautwein Christian, Strnad Pavel
Abstract excerpt
BACKGROUND & AIMS: α1-Antitrypsin (AAT) is a major protease inhibitor produced by hepatocytes. The most relevant AAT mutation giving rise to AAT deficiency (AATD), the 'Pi∗Z' variant, causes harmful AAT protein accumulation in the liver, shortage of AAT in the systemic circulation, and thereby predisposes to liver and lung injury. Although intravenous AAT augmentation constitutes an established treatment of...
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